| ALL TIMES ARE IN EASTERN TIME ZONE | |||||
| Start | Finish | Topic/Event | Speaker | ||
| Tuesday, July 18th | |||||
| Medical Symposium, Day One | Moderator: Dr. Robert Thompson Stone | ||||
| 9:00am | 9:05am | Welcome | |||
| 9:05am | 9:35am | Krabbe Disease & RUSP History | Dr. Dietrich Matern | ||
| 9:35am | 9:50am | Krabbe Newborn Screening Council | Dr. Joanne Kurtzberg | ||
| State NBS Updates Panel | |||||
| 9:50am | 10:00am | New York | Dr. Joseph Orsini | ||
| 10:00am | 10:10am | Illinois | Dr. Joshua Baker | ||
| 10:10am | 10:20am | Tennessee | Gwendolyn McKee | ||
| 10:20am | 10:30am | Ohio | Dr. Margie Ream | ||
| 10:30am | 10:40am | Pennsylvania | Pat Edge | ||
| 10:40am | 10:45am | Kentucky | Dr. Dietrich Matern | ||
| 10:45am | 10:50am | Indiana | Jennifer Weaver | ||
| 10:50am | 10:55am | Georgia | Dr. Stephanie Keller | ||
| 10:55am | 11:00am | New Jersey | Caitlin Russo | ||
| 11:00am | 11:05am | South Carolina | Beth Bair | ||
| 11:05am | 11:35am | Update on HSCT for Krabbe Disease | Dr. Joanne Kurtzberg | ||
| 11:35am | 12:05pm | Update on Gene Therapy for Krabbe Disease | Dr. Maria Escolar | ||
| 12:05pm | 12:20pm | Discussion | |||
| 1:30pm | 2:30pm | NBS Positive for Krabbe Disease - What now? | Dr. Robert Stone | ||
| 2:30pm | 3:00pm | Panel Discussion of NBS for Krabbe Disease: Sensitivity vs. Specificity, Cure vs. Chance of Life | Dr. Dietrich Matern | ||
| 3:00pm | 3:30pm | Update on Newborn Screening for ALD | Dr. Joshua Bonkowsky | ||
| 3:30pm | 3:45pm | Update on NBS for MLD | Dr. Michael Gelb | ||
| 3:45pm | 4:00pm | Update on NBS for CTX | Dr. Michael Gelb | ||
| Wednesday, July 19th | |||||
| Medical Symposium, Day Two | Moderator: Dr. Jennifer Rubin | ||||
| Leukodystrophy Care Network | |||||
| 8:00am | 9:00am | LCN Phase II - Current gaps and unmet needs in the Leukodystrophy Community | Anna Grantham | ||
| 9:00am | 9:20am | Transitioning into Adulthood: Pitfalls and Opportunities | Catie Becker | ||
| 9:20am | 9:50am | Musculoskeletal CPG | Dr. Sally Evans | ||
| 9:50am | 10:20am | Pulmonary CPG | Dr. Geovanny Perez | ||
| 10:20am | 10:50am | The genetics of cerebrotendinous xanthomatosis and implications for NBS | Dr. Austin Larson | ||
| Updates on Treatments and Clinical Trials for Leukodystrophies | |||||
| 10:50am | 11:20am | DUOC: A Cord Blood Therapy to Myelinate the Brain | Dr. Joanne Kurtzberg | ||
| 11:20am | 11:35am | A Clinical Trial of Leriglitazone in Adult Cerebral Adrenoleukodystrophy | Dr. Ali Fatemi | ||
| 11:35am | 12:00pm | Discussion | |||
| 12:00pm | 12:45pm | Lunch | |||
| 12:45pm | 1:15pm | Orchard Therapeutics | Angela Tom | ||
| 1:15pm | 1:45pm | Vigil Therapeutics - CSF1R | Dr. Andreas Meier | ||
| 1:45pm | 2:15pm | Clinical Trials in the Ultra Rare Leukodystrophies | Dr. Amanda Nagy | ||
